FDA Center of Excellence and the Drug Approval Process

Joseph Alvarnas, MD: You actually raised this question earlier by saying there’s limited competition, actually virtually no competition, for newer agents. As we think about some of the dimensions of increasing the potential for competition, the FDA Safety and Innovation Act allows the FDA to recognize drug inspections conducted by foreign regulatory authorities. Two questions: What are your thoughts on drug imports as a way of increasing competition? But beyond that, with hopefully lower cost generics and more meaningful biosimilars and increasing numbers of biosimilars, can we hope for a race to the bottom with prices?

John Fox, MD: I’m from Michigan, and we have lots of people going across the border to buy their drugs. I can’t pay for them, but I think opening access to imported drugs would have a tendency to drive down prices. And at least—especially for patients who are paying first dollar for their drugs, because they have a significant deductible; I’m not sure this will apply in the cancer space, but for certainly other drugs— importation of drugs would allow them to have a more affordable option than they have today.

Elizabeth Carpenter: From a policy perspective, this is another idea that’s getting some bipartisan traction on Capitol Hill. Obviously, members of Congress have debated importation for decades or longer at this point. But it is re-emerging, and there are some Republicans who have proved to be supportive, and certainly there are members on the Democratic side. Obviously, the same issues continue to be raised. Is this safe? Is this practical from an operational standpoint?

And I go back to this idea: There’s a spectrum of how we could take some of these steps. On one hand, you have importation of every medication, which is likely more challenging for a number of reasons. On the other hand, you have unique instances where, again, a medication is off-patent, they don’t have a competitor, and they’re taking a price increase that members of the Congress or the administration deems unreasonable. That could trigger some kind of importation authority, and that’s something that we’ve seen members really on both sides of the aisle start to talk about.

Joseph Alvarnas, MD: Wonderful. To add a different layer to this, the Trump Administration has not only articulated that drugs need to be less expensive, but that they also need to come to the market faster. Bob, what are your thoughts about the FDA’s Oncology Center of Excellence and its role in accelerating the process of bringing drugs to the market?

Robert Carlson, MD: First of all, that Center of Excellence is recognizing that oncology is different than other specialties and the oncology drugs have different criteria for approval, different magnitudes of toxicity, and a different skill set for what it takes to evaluate them appropriately from a regulatory perspective. They set up the Center of Excellence within the FDA, so it’s oncology within the FDA. Richard Pazdur is the founding director of that Center of Excellence and a wonderful choice. He’s got incredible experience within the FDA and oncology evaluations. He is trusted by all the stakeholders that are involved. I expect great things out of that—under his leadership.

I would hope that that sort of Center of Excellence is going to accelerate the review of drugs within the FDA. The speed with which drugs are currently—at least oncologic drugs are—evaluated within the FDA is actually quite fast. I think it’s under 1 year now—11 months was the last figure that I saw, which was remarkably fast. There is some room to go, but not a lot if we’re going to do it in a safe and appropriate scientific way. But I would hope that the bandwidth in terms of the numbers of drugs that they can look at will increase.

John Fox, MD: To be clear, Bob, we’re talking about accelerating the review of the data that have been submitted and required by the FDA. We’re not talking about reviewing or requiring less information in order to get approval. Or are we talking both?

Robert Carlson, MD: I think primarily we’re talking about more rapid approval. There may be some circumstances where they require less data for approval. There’s a lot of discussion. We’ve heard some of the discussion now at the national level in terms of whether or not all we should look at is safety, and once something is safe, it doesn’t matter if it’s effective. It should be approved and available. Now I actually tend to believe that, if something has no efficacy, any toxicity is an infinite toxicity in terms of…

John Fox, MD: Harm.

Robert Carlson, MD: In terms of harm, and so I’m not willing to buy into that. I think you first have to show effectiveness, and then you have to show safety. Hopefully, you’ll show both together. But I do think the approval process is likely to accelerate, and hopefully that will also allow the FDA to focus more attention on defining what is required to get a drug approved—so that it’s more clear what endpoints they’re willing to accept, what the trade-offs between toxicity and efficacy are, and those sorts of considerations.

John Fox, MD: I think the concern that I have—and we should all have, I think—is that the desire to get drugs to market faster and faster results in less and less information. I think as a physician, and you as an oncologist, you want to know what optimal therapy is for your patient. And with some of these accelerated approval pathways for drugs that are approved on a single phase II, non-randomized clinical trial, do we have enough safety data to understand what the options are for that patient?

There was just a study published in JAMA’s Internal Medicine in December of last year that showed that 18 drugs that had been approved had no survival benefit, and that 17 of those 18 either had no improvement in quality of life or a worsened quality of life. And yet, we approved them all. All of them except one are still on the market. So, I’m concerned not only that we’re approving drugs without adequate information to make an informed decision—either the patient or the provider or both—about what the best therapy is for that patient, but that we’re also expending extraordinary dollars for therapy that may have no benefit. And that’s a cost to all of us, not only as a payer or the employers that we serve but to society as a whole.

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